On September 10, 2019 The New York Proton Center (NYPC) reported that has treated its first proton therapy cancer patients (Press release, Varian Medical Systems, SEP 10, 2019, View Source [SID1234539405]). Equipped with the Varian ProBeam proton therapy system, NYPC is New York State’s first proton therapy facility and is a collaboration between Memorial Sloan Kettering Cancer Center, Montefiore Health System, and Mount Sinai Health System .

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NYPC is expected to treat 1,400 patients annually, including most children in New York City with solid tumors, delivering the most advanced form of cancer radiation treatment currently available. Before NYPC was opened, proton therapy patients in the New York Metro area were referred to out-of-state facilities.

"We are thrilled to be delivering the highest standard of treatment possible and ensuring our patients – many of them children – are receiving this advanced oncology therapy without having to travel out of the state," said Dr. Charles B. Simone,II, FACRO, chief medical officer at the New York Proton Center. "With the state-of-the-art Varian ProBeam system, we know that our experienced team of proton therapy experts are able to provide patients with the most advanced radiation treatment modality possible."

Proton therapy uses protons, accelerated to about two-thirds the speed of light, or more than 100,000 miles per second, to destroy cancer cells, while minimizing exposure to nearby healthy tissues. NYPC will treat pediatric and adult patients with a wide variety of conditions, including brain and spine tumors, head and neck tumors, breast cancer, lung and other thoracic cancers, gastrointestinal cancers, sarcomas, gynecologic cancers, prostate cancer, lymphomas and recurrence cancers.

"Seeing our collaboration with long-term strategic partners in radiation oncology at New York Proton Center come to fruition is a very proud moment for Varian," said Kolleen Kennedy, president, Proton Solutions and chief growth officer at Varian. "This means the most advanced cancer care for patients in the greater New York area, and we are excited to build upon this strong foundation as we partner to deliver on the promise of a world without fear of cancer."

On September 10, 2019 Kindred Biosciences, Inc. (NASDAQ: KIN), a commercial-stage biopharmaceutical company developing novel drugs and biologics, reported that it has been selected by the National Cancer Institute (NCI) as one of three contractors in response to the solicitation for the PREVENT Cancer Preclinical Drug Development Program (PREVENT): Current Good Manufacturing Practice (cGMP) Production of Vaccines and Biologicals for Cancer Prevention (cGMP Pool) (Press release, Kindred Healthcare, SEP 10, 2019, View Source [SID1234539421]). As a cGMP pool contractor, KindredBio is eligible to provide manufacturing, formulation and analytical services to meet the needs of the PREVENT pipeline.

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PREVENT is an NCI-supported venture to advance preclinical development of innovative cancer prevention interventions and biomarkers towards clinical applications. A maximum amount of $49.95 million will be shared across three contract pools, one of which is the cGMP manufacturing pool. Within the cGMP pool, KindredBio is one of three contractors selected for award. The contract term is four years, with specific amounts based on individual task order awards yet to be determined.

"This selection is a validation of our manufacturing and project management expertise, and we look forward to working with the NCI on this important program," said Richard Chin, CEO of KindredBio.

KindredBio operates a state-of-the-art biological development and cGMP manufacturing facility in Burlingame, CA, and recently completed construction on a 180,000 square foot large scale cGMP manufacturing facility in Elwood, KS.

The company’s core expertise includes protein engineering, cell line development, master cell banking, process development, assay development, stability testing, and cGMP clinical and commercial manufacturing. KindredBio’s biologics team comprises experts in product development, manufacturing, quality control and quality assurance personnel, and is supported by a strong project management organization.

For additional information on PREVENT, please click here. Task orders awarded to KindredBio are expected to be conducted at the company’s cGMP manufacturing facility in Burlingame, CA.

This project has been funded in whole or in part with Federal funds from the National Cancer Institute, National Institutes of Health, Department of Health and Human Services, under Contract No. 75N91019D00027.

On September 10, 2019 Oncoceutics, Inc. reported a scientific publication in the Journal of Neuro-Oncology describing a complete response and other forms of clinical benefit in pediatric and adult H3 K27M-mutant glioma patients treated with ONC201 on expanded access protocols (Press release, Oncoceutics, SEP 10, 2019, View Source [SID1234558332]).

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This manuscript details radiographic findings and other clinical outcomes in eighteen H3 K27M-mutant glioma patients who were enrolled on expanded access protocols. These patients initiated ONC201 after prior radiation, and in many cases after also failing additional chemotherapy, a disease setting in neuro-oncology where no available therapies have been proven effective.

Overall findings include disease stabilization and radiographic regressions after initiation of ONC201 that are atypical for the disease course. Two adult and two pediatric cases are highlighted due to improvements in radiographic imaging of their disease, as well as disease-associated neurological symptoms. The most dramatic response was observed in an adult who initiated ONC201 at second recurrence following prior radiation and chemotherapy. After initiating ONC201, the patient’s tumor located in the thalamus and other sites in the brain completely regressed. These imaging findings were accompanied by improvements in headaches, nausea, and right-sided numbness caused by the disease.

Among the 14 patients with recurrent disease prior to initiation of ONC201, median progression-free survival was 14 weeks and median overall survival was 17 weeks. Three adults among the 14 recurrent patients remain on treatment progression-free with a median follow up of 41, 49.6, and 76.1 weeks. Among the 4 pediatric patients who initiated adjuvant ONC201 following radiation, two DIPG patients remain progression-free for at least 53 and 81 weeks and the other two progressed at 28.4 and 41.9 weeks.

"Expanded access to ONC201 was critical in allowing our H3 K27M-mutant patients to access the therapy while clinical trials were being initiated for this form of glioma that previously had no effective therapy," said Nicole Shonka, MD, Associate Professor of Internal Medicine at Nebraska Medical Center. "The complete response in the patient I treated is very striking, given the dismal prognosis of this type of grade IV glioma that is typically unresponsive to anything after initial radiation. We are eager to continue the evaluation of ONC201 in clinical trials to further establish the exciting results we saw in expanded access."

On September 10, 2019 University of California, reported the U.S. Patent and Trademark Office (USPTO) granted a new CRISPR-Cas9 patent to the University of California (UC), University of Vienna, and Dr. Emmanuelle Charpentier covering single-molecule guide RNAs or nucleic acid molecules encoding the guide RNAs.

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Today’s patent (U.S. 10,407,697) is the 13th in the university’s U.S. CRISPR-Cas9 portfolio and follows the issuance of 10 other patents in the past six months. Continuing the momentum into the fall, UC expects to further expand its portfolio through the issuance of four additional patents in the near future. In total, the patents that have issued and those that are set to issue bring the university’s portfolio to 17 U.S. CRISPR-Cas9 patents covering various compositions and methods of targeting and editing genes in any setting, such as within plant, animal, and human cells, as well as modulating transcription.

"We are pleased by our recent trajectory that has significantly increased UC’s intellectual property protection of the Doudna-Charpentier team’s work on CRISPR-Cas9," said Eldora L. Ellison, Ph.D., lead patent strategist on CRISPR-Cas9 matters for UC and a Director at Sterne, Kessler, Goldstein & Fox. "We will continue pursuing claims for this pioneering gene-editing technology so we can ensure the methods are utilized for the benefit of society."

The Doudna-Charpentier team that invented the CRISPR-Cas9 DNA-targeting technology included Jennifer Doudna and Martin Jinek at the University of California, Berkeley; Emmanuelle Charpentier (then of Umea University); and Krzysztof Chylinski at the University of Vienna. The single-molecule guide RNAs covered by today’s patent, as well as the other compositions and methods claimed in UC’s previously issued patents and those set to issue, were included among the CRISPR-Cas9 gene editing technology work disclosed first by the Doudna-Charpentier team in its May 25, 2012 priority patent application.

Additional CRISPR-Cas9 patents in this team’s portfolio include 10,000,772; 10,113,167; 10,227,611; 10,266,850; 10,301,651; 10,308,961; 10,337,029; 10,351,878; 10,358,658; 10,358,659; 10,385,360; and 10,400,253. These patents are not a part of the PTAB’s recently declared interference between 14 UC patent applications and multiple previously issued Broad Institute patents and one application, which jeopardizes essentially all of the Broad’s CRISPR patents involving eukaryotic cells.

International patent offices have also recognized the pioneering innovations of the Doudna-Charpentier team, in addition to the 13 patents granted in the U.S. so far. The European Patent Office (representing more than 30 countries), as well as patent offices in the United Kingdom, China, Japan, Australia, New Zealand, Mexico, and other countries, have issued patents for the use of CRISPR-Cas9 gene editing in all types of cells.

University of California has a long-standing commitment to develop and apply its patented technologies, including CRISPR-Cas9, for the betterment of humankind. Consistent with its open-licensing policies, UC allows nonprofit institutions, including academic institutions, to use the technology for non-commercial educational and research purposes.

In the case of CRISPR-Cas9, UC has also encouraged widespread commercialization of the technology through its exclusive license with Caribou Biosciences, Inc. of Berkeley, California. Caribou has sublicensed this patent family to numerous companies worldwide, including Intellia Therapeutics, Inc. for certain human therapeutic applications. Additionally, Dr. Charpentier has licensed the technology to CRISPR Therapeutics AG and ERS Genomics Limited.

On September 10, 2019 VBI Vaccines Inc. (Nasdaq: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported a collaboration with GlaxoSmithKline (GSK) to clinically evaluate the combination of VBI-1901, VBI’s cancer vaccine immunotherapeutic, with GSK’s proprietary AS01B adjuvant system (Press release, VBI Vaccines, SEP 10, 2019, View Source [SID1234539406]). As part of the collaboration, VBI plans to add an additional study arm to Part B of the company’s ongoing, multi-center, open-label Phase 1/2a clinical study targeting recurrent glioblastoma (GBM), a cytomegalovirus (CMV)-associated tumor.

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"VBI-1901 has shown encouraging results in Part A of the ongoing Phase 1/2a clinical study in recurrent GBM patients and we are excited to be able to expand the scope of Part B to assess the candidate in combination with AS01B, a highly-innovative adjuvant system that has contributed to positive results in combination with the gE antigen in GSK’s shingles vaccine, Shingrix," said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer. "VBI’s enveloped virus-like particle (eVLP) technology, the basis for VBI-1901, is highly versatile and has demonstrated clinical potency in both preventative and therapeutic settings. We believe that these two technologies may be an ideal match for next-generation vaccines, and we look forward to seeing the results of this collaboration."

"This is the first time we have partnered with a biopharma company to evaluate AS01B in such a clinical setting and the first time this adjuvant will be assessed in oncology for GBM patients. We have shown the ability of AS01B to boost T-cell mediated immunity and believe the combination of AS01B and VBI-1901 could have benefits for patients with glioblastoma, a rare but devastating cancer," said Emmanuel Hanon, Senior Vice President, Head of R&D at GSK Vaccines.

In Part A of the study, VBI-1901 adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF) was well-tolerated at all doses. Further, three out of six patients in the high-dose (10 µg) cohort demonstrated evidence of stable disease by magnetic resonance imaging (MRI), which correlated with vaccine-induced immune response. Based on this safety and immunogenicity data, the high-dose was identified as the optimal therapeutic dose to test in the Part B extension phase of the study.

Part B of the ongoing Phase 1/2a clinical study is now planned to be a two-arm, open-label study, enrolling 20 first-recurrent GBM patients to receive VBI-1901 in combination with either GM-CSF or AS01B as immunomodulatory adjuvants. Enrollment of the 10 patients in the VBI-1901 with GM-CSF arm was initiated at the end of July 2019. Initiation of enrollment of the 10 patients in the VBI-1901 with AS01B arm is expected later in the second half of 2019, subject to U.S. Food and Drug Administration (FDA) acceptance of the amended protocol.

VBI’s ongoing two-part study is being conducted at The Neurological Institute of New York Columbia University Medical Center, Dana-Farber Cancer Institute, and Massachusetts General Hospital.

About the Phase 1/2a Study Design

VBI’s two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in up to 38 patients with recurrent GBM:

Part A:

Dose-escalation phase that defined the safety, tolerability, and optimal dose level of VBI-1901 in recurrent GBM patients, with any number of prior recurrences
This phase enrolled 18 recurrent GBM patients across three dose cohorts of VBI-1901: 0.4 µg, 2.0 µg, and 10.0 µg
Enrollment completed in December 2018

Part B:

Subsequent extension of the optimal dose level, 10.0 µg, as defined in the Part A dose escalation phase
This phase will be a two-arm study, enrolling 10 patients in each arm, assessing VBI-1901 in combination with either GM-CSF or AS01B as immunomodulatory adjuvants
Part B will enroll first-recurrent GBM patients only

VBI-1901 is administered intradermally when adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF), and will be administered intramuscularly when adjuvanted with GSK proprietary AS01B adjuvant system. Patients in both phases of the study will receive the vaccine immunotherapeutic every four weeks until clinical progression.

Additional information, including a detailed description of the study design, eligibility criteria, and investigator sites, is available at ClinicalTrials.gov using identifier NCT03382977.

About Glioblastoma (GBM)

Scientific literature suggests cytomegalovirus (CMV) infection is prevalent in multiple solid tumors, including GBM, gliomas, and breast cancer, among others. GBM is among the most common and aggressive malignant primary brain tumors. In the U.S. alone, 12,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and is exceptionally lethal.